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IP Update: Recent Patents, Patent Applications Awarded to Alnylam, Novartis, Tekmira, and More

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Title: Vaccine Compositions and Methods for Treatment of Mucormycosis and Other Fungal Diseases

Patent Number: 8,444,985

Filed: March 19, 2010

Lead Inventor: Ashraf Ibrahim, Los Angeles Biomedical Research Institute

The invention, the patent’s abstract states, comprises “therapeutic compositions and methods for treating and preventing fungal disease or conditions including mucormycosis. The therapeutic methods and compositions of the invention include vaccine compositions having an FTR polypeptide or an antigenic fragment of the polypeptide; a vector including a nucleotide sequence that is substantially complimentary to at least 18 contiguous nucleotides of FTR sequence; an antisense; a small interfering RNA; or an antibody inhibitor of FTR. The vaccine compositions of the invention can further include an adjuvant.”


Title: RNA Interference-Mediating Small RNA Molecules

Patent Number: 8,445,237

Filed: July 12, 2010

Lead Inventor: Thomas Tuschl, Max Planck Institute

“Double-stranded RNA induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference,” the patent’s abstract states. “Using a Drosophila in vitro system, we demonstrate that 19-23 [nucleotide] short RNA fragments are the sequence-specific mediators of RNAi. The short interfering RNAs are generated by an RNase III-like processing reaction from long dsRNA. Chemically synthesized siRNA duplexes with overhanging 3' ends mediate efficient target RNA cleavage in the lysate, and the cleavage site is located near the center of the region spanned by the guiding siRNA. Furthermore, we provide evidence that the direction of dsRNA processing determines whether sense or antisense target RNA can be cleaved by the produced siRNP complex.”


Title: Snap-Back Primers and Detectable Hairpin Structures

Patent Number: 8,445,238

Filed: Oct. 3, 2007

Lead Inventor: Jeff Hall, Third Wave Technologies

The invention provides “methods, compositions, and kits comprising snap-back primers used for forming 3' hairpin structures, 5' hairpin structures, and double hairpin structures,” the patent’s abstract states. “The hairpin structures may be used for detecting target sequences, for detecting polymorphisms in target sequences, or other nucleic acid characterization methods. In certain embodiments, the hairpin structures form invasive cleavage structures, which may be cleaved by structure-specific enzymes in order to detect the presence or absence of a particular nucleotide or nucleotide sequence.”


Title: Tyrosine-modified Recombinant rAAV Vector Compositions and Methods for Use

Patent Number: 8,445,267

Filed: April 8, 2008

Lead Inventor: Li Zhong, University of Florida

The invention comprises “tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations,” the patent’s abstract states. “Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.”


Title: Use of Minicircle Vectors for Cardiac Gene Therapy

Patent Number: 8,445,454

Filed: Oct. 22, 2010

Lead Inventor: Joseph Wu, Stanford University

The patent, its abstract states, claims “compositions and methods … for the treatment of an ischemic cardiovascular condition by providing a patient with a novel non-viral minicircle DNA vector comprising polynucleotide sequences that potentiate HIF-1 activity, including RNAi or antisense agents selective for proteins involved in HIF1 inactivation.”


Title: Use of RNA Interference for Treating or Reducing Pain

Patent Number: 8,445,456

Filed: July 14, 2011

Lead Inventor: Ming-Dar Tsai, Fu-Jen Catholic University

The invention, the patent’s abstract states, comprises “a nucleic acid molecule mediating RNA interference for treating or reducing pain. The nucleic acid molecule … effectively inhibit[s] expression of bradykinin B2 receptor.”


Title: Therapeutic Compositions

Patent Number: 8,445,665

Filed: April 6, 2010

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The patent, its abstract states, “relates to iRNA agents and methods of making and using the agents. The iRNA agent comprises a sense sequence and an antisense sequence … [and] can present increased nuclease resistance.”


Title: Ribonucleic Acid Interference Molecules

Patent Number: 8,445,666

Filed: Oct. 27, 2011

Lead Inventor: Isidore Rigoutsos, IBM

The patent, its abstract states, claims a method for regulating gene expression via RNAi using a nucleic acid molecule with one of one or more predefined precursor sequences.


Title: siRNA Targeting Cyclin-dependent Kinase Inhibitor 1B

Patent Number: 8,445,667

Filed: June 25, 2012

Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)

“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” the patent’s abstract states. “By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for CDKN1B.”


Title: siRNA Targeting Apolipoprotein B

Patent Number: 8,445,668

Filed: June 28, 2012

Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)

“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” the patent’s abstract states. “By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for apoB.”


Title: microRNA-198 as a Tumor Suppressor in Pancreatic Cancer

Application Number: 20130121912

Filed: Aug. 6, 2012

Lead Inventor: Qizhi Yao, Baylor College of Medicine

The invention, the patent application’s abstract states, is based on the identification of “a novel network of tumorigenic prognostic factors … that plays a critical role in advanced pancreatic cancer pathogenesis. This interactome is interconnected through a central tumor suppressive microRNA, miR-198, which is able to both directly and indirectly modulate expression of the various members of this network to alter the molecular makeup of pancreatic tumors. When this tumor signature network is intact, miR-198 expression is reduced and patient survival is dismal; patients with higher miR-198 present an altered tumor signature network, better prognosis and increased survival. Further, MiR-198 replacement reverses tumorigenicity in vitro and in vivo.” The invention relates to “a method of treating cancer in an individual [by] increasing the level of active microRNA-198 molecules in the pancreatic cancer tumor cell … to cause an improvement in the pancreatic cancer in the individual.”


Title: Poly(vinyl ester) Polymers for In Vivo Nucleic Acid Delivery

Application Number: 20130121954

Filed: Aug. 23, 2012

Lead Inventor: Darren Wakefield, Arrowhead Research

The invention, the patent application’s abstract states, comprises “membrane active poly(vinyl ester) polymers and compositions for targeted delivery of RNA interference polynucleotides to cells in vivo. RNAi polynucleotides are conjugated to the poly(vinyl ester) polymers and the polymers are reversibly modified to enable in vivo targeted delivery. Membrane activity of the poly(vinyl ester) provides for movement of the RNAi polynucleotides from outside the cell to inside the cell. Reversible modification provides physiological responsiveness.”


Title: Compositions for Drug Delivery and Methods of Manufacturing and Using the Same

Application Number: 20130122096

Filed: Nov. 14, 2012

Lead Inventor: Amotz Shemi, Silenseed

The patent application, its abstract states claims “polymeric implants containing microparticles and nanoparticles, [and] delivery of microparticles and nanoparticles from a polymeric implant.”


Title: Novel Lipid Formulations for Delivery of Therapeutic Agents to Solid Tumors

Application Number: 20130122104

Filed: Aug. 3, 2012

Lead Inventor: Ed Yaworski, Protiva Biotherapeutics (Tekmira Pharmaceuticals)

The invention, the patent application’s abstract states, “provides novel, serum-stable lipid particles comprising one or more active agents or therapeutic agents, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles. More particularly, the present invention provides serum-stable nucleic acid-lipid particles comprising a nucleic acid, methods of making the SNALP, and methods of delivering and/or administering the SNALP. In particular embodiments, the … invention provides tumor-directed lipid particles that preferentially target solid tumors. The tumor-directed formulations of the present invention are capable of preferentially delivering a payload such as a nucleic acid to cells of solid tumors compared to non-cancerous cells.”


Title: Dual Targeted siRNA Therapeutics for Treatment of Diabetic Retinopathy and Other Ocular Neovascularization Diseases

Application Number: 20130123330

Filed: July 15, 2012

Lead Inventor: Patrick Lu, Sirnaomics

The invention relates to “compositions and methods for treating diabetic retinopathy and other ocular neovascularization diseases,” according to the patent application’s abstract. “In one embodiment, the composition comprises at least two different siRNA duplexes and a pharmaceutically acceptable carrier. One of the duplexes binds to an mRNA molecule that encodes VEGF, and the other binds to an mRNA molecule that encodes VEGFR2. In another embodiment, the composition further comprises an siRNA duplex that binds to an mRNA molecule that encodes TGF-beta1.”


Title: Compositions and Methods for Inhibiting Expression on Mylip/Idol Gene

Application Number: 20130123332

Filed: Jan. 25, 2011

Lead Inventor: Brian Bettencourt, Alnylam Pharmaceuticals

The invention, the patent application’s abstract states, “relates to double-stranded ribonucleic acid compositions targeting the Mylip/Idol gene, and methods of using such dsRNA compositions to inhibit expression of Mylip/Idol.”


Title: Nucleic Acid Functionalized Nanoparticles for Therapeutic Applications

Application Number: 20130123333

Filed: Aug. 20, 2012

Lead Inventor: Chad Mirkin Northwestern University (AuraSense Therapeutics)

The patent application, its abstract states, claims “materials and methods for regulating gene expression using nanoparticles functionalized with antisense oligonucleotides.”


Title: Novel siRNA Structures

Application Number: 20130123334

Filed: Aug. 27, 2012

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

The invention relates to “siRNA compounds possessing novel sequences and structural motifs [that] downregulate the expression of specific human genes,” the patent application’s abstract states. “The invention also relates to pharmaceutical compositions comprising such compounds and a pharmaceutically acceptable carrier … [as well as] a method of treating and/or preventing the incidence or severity of various diseases or conditions associated with the genes and/or symptoms associated with such diseases or conditions comprising administering to a subject in need of treatment for such disease or condition and/or symptom the compound or the pharmaceutical composition in a therapeutically effective dose so as to thereby treat the subject.”


Title: Polyplexes of Hydrophobically Modified siRNA for Delivery of siRNA

Application Number: 20130123336

Filed: Nov. 1, 2012

Lead Inventor: Joseph Vetro, University of Nebraska

The invention provides “compositions and methods for delivering nucleic acid molecules to a cell,” the patent application’s abstract states.


Title: RNAi Inhibition of Serum Amyloid A for Treatment of Glaucoma

Application Number: 20130123337

Filed: Nov. 20, 2012

Lead Inventor: Abbot Clark, Novartis

“RNA interference is provided for inhibition of serum amyloid A mRNA expression in glaucoma involving SAA expression,” the patent application’s abstract states.


Title: Novel Cationic Lipids and Methods of Use Thereof

Application Number: 20130123338

Filed: May 12, 2011

Lead Inventor: James Heyes, Protiva Biotherapeutics (Tekmira Pharmaceuticals)

The invention, the patent application’s abstract states, provides “compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel cationic lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells in vivo. The compositions of the … invention are highly potent, thereby allowing effective knockdown of a specific target protein at relatively low doses. In addition, the compositions and methods of the … invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.”


Title: Compositions and Methods for Silencing Apolipoprotein B

Application Number: 20130123339

Filed: May 12, 2011

Lead Inventor: James Heyes, Protiva Biotherapeutics (Tekmira Pharmaceuticals)

The invention provides “compositions and methods for the delivery of interfering RNAs … that silence apoB expression in cells such as liver cells,” the patent application’s abstract states. “In particular, the nucleic acid-lipid particles provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic acid to cells such as liver cells in vivo.”


Title: Methods and Compositions for the Specific Inhibition of KRAS by Asymmetric Double-Stranded RNA

Application Number: 20130123342

Filed: Jan. 10, 2013

Inventor: Bob Brown, Dicerna Pharmaceuticals

The invention, the patent application’s abstract states, relates to “compounds, compositions, and methods useful for reducing KRAS target RNA and protein levels via use of Dicer-substrate siRNA agents possessing asymmetric end structures.”

The Scan

Pfizer-BioNTech Seek Full Vaccine Approval

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Viral Integration Study Critiqued

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Giraffe Species Debate

The Scientist reports that a new analysis aiming to end the discussion of how many giraffe species there are has only continued it.

Science Papers Examine Factors Shaping SARS-CoV-2 Spread, Give Insight Into Bacterial Evolution

In Science this week: genomic analysis points to role of human behavior in SARS-CoV-2 spread, and more.